Research Articles - Other Retinal Diseases
On the heels of the report in The Lancet that choroideremia gene therapy restored vision in a human study at Oxford University, a new company called Nightstar has been spun out of the institution to advance the treatment as well as other emerging retinal disease gene therapies. Syncona LLP, an independent subsidiary of the Wellcome Trust, which is funding the choroideremia trial, is making an investment of more than $16 million in Nightstar.
Aerobic exercise is well-known for helping people stay trim, stave off heart disease and improve mood, but a study by researchers at Emory University and the Atlanta Veterans Administration Medical Center suggests that it also can preserve vision in those with retinal degenerative diseases. While the investigation was in mice, the scientists believe the results, published in The Journal of Neuroscience, may translate to humans.
In a research paper recently published in the Proceedings of the National Academy of Sciences, investigators from the University College of London (UCL) reported on their successful integration of stem cell transplants in six mouse models of retinal disease, including those for retinitis pigmentosa (RP), Leber congenital amaurosis and congenital stationary night blindness (CSNB).
The Children’s Hospital of Philadelphia (CHOP) is investing $50 million in Spark Therapeutics, its newly formed spinoff company, to lead its landmark gene-therapy clinical trial for Leber congenital amaurosis (LCA, RPE65 mutations), an inherited form of blindness affecting children, and hemophilia B, an inherited blood-clotting disorder. The company also plans to advance additional gene therapies, including those for other
Drs. Albert Maguire, Katherine High and
Jean Bennett of CHOP
A Foundation-funded research team led by investigators from the University of Pennsylvania has reported that its emerging gene therapy for Best disease, a form of inherited macular degeneration, has cleared all the vision-robbing lesions in canines with the condition. Success in the study moves scientists closer to a human study of the treatment. Dr. Karina Guziewicz in collaboration with
Ocular Regeneration: Cell Therapy and Regeneration in the Retina
Presented in partnership with the Third International Biennial Symposium on AMD*
Thursday, October 23, 2014
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Foundation-funded researchers at the Radboud University Medical Center in the Netherlands have found that mutations in the gene POC1B can cause cone dystrophy (COD) and cone-rod dystrophy (CRD). These retinal degenerative conditions are characterized by progressive loss of central vision, with CRD also leading to peripheral vision loss. The discovery of harmful defects in POC1B will help scientists diagnose more